Ferret and Pig Models of Cystic Fibrosis: Prospects and Promise for Gene Therapy

Ziying Yan; Zoe A Stewart; Patrick L Sinn; John C Olsen; Jim Hu; Paul B McCray; John F Engelhardt

doi:10.1089/humc.2014.154

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Ferret and Pig Models of Cystic Fibrosis: Prospects and Promise for Gene Therapy

Journal article

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Ferret and Pig Models of Cystic Fibrosis: Prospects and Promise for Gene Therapy

Ziying Yan, Zoe A Stewart, Patrick L Sinn, John C Olsen, Jim Hu, Paul B McCray and John F Engelhardt

Human gene therapy. Clinical development, Vol.26(1), pp.38-49

03/01/2015

DOI: 10.1089/humc.2014.154

PMCID: PMC4367511

PMID: 25675143

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Abstract

Large animal models of genetic diseases are rapidly becoming integral to biomedical research as technologies to manipulate the mammalian genome improve. The creation of cystic fibrosis (CF) ferrets and pigs is an example of such progress in animal modeling, with the disease phenotypes in the ferret and pig models more reflective of human CF disease than mouse models. The ferret and pig CF models also provide unique opportunities to develop and assess the effectiveness of gene and cell therapies to treat affected organs. In this review, we examine the organ disease phenotypes in these new CF models and the opportunities to test gene therapies at various stages of disease progression in affected organs. We then discuss the progress in developing recombinant replication-defective adenoviral, adeno-associated viral, and lentiviral vectors to target genes to the lung and pancreas in ferrets and pigs, the two most affected organs in CF. Through this review, we hope to convey the potential of these new animal models for developing CF gene and cell therapies.

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Details

Title: Subtitle: Ferret and Pig Models of Cystic Fibrosis: Prospects and Promise for Gene Therapy
Creators: Ziying Yan - 2Center for Gene Therapy of Cystic Fibrosis, University of Iowa School of Medicine, Iowa City, IA 52242
Zoe A Stewart - 3Department of Surgery, University of Iowa School of Medicine, Iowa City, IA 52242
Patrick L Sinn - 4Department of Pediatrics, University of Iowa School of Medicine, Iowa City, IA 52242
John C Olsen - 5Cystic Fibrosis/Pulmonary Research and Treatment Center, Department of Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599
Jim Hu - 6Department of Laboratory Medicine and Pathobiology, Hospital for Sick Children and University of Toronto, Toronto, ON M5G 0A4, Canada
Paul B McCray - 4Department of Pediatrics, University of Iowa School of Medicine, Iowa City, IA 52242
John F Engelhardt - 7Department of Internal Medicine, University of Iowa School of Medicine, Iowa City, IA 52242
Resource Type: Journal article
Publication Details: Human gene therapy. Clinical development, Vol.26(1), pp.38-49
DOI: 10.1089/humc.2014.154
PMID: 25675143
PMCID: PMC4367511
NLM abbreviation: Hum Gene Ther Clin Dev
ISSN: 2324-8637
eISSN: 2324-8645
Publisher: Mary Ann Liebert, Inc
Language: English
Date published: 03/01/2015
Academic Unit: Roy J. Carver Department of Biomedical Engineering; Microbiology and Immunology; Pulmonary Medicine; Anatomy and Cell Biology; Stead Family Department of Pediatrics; Radiation Oncology; Internal Medicine
Record Identifier: 9984025453502771

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